A long-term study on changes in height and weight of children with MPS II receiving Elaprase and who started the treatment before the age of 6 years.

Phase 1

• Conditions: Hunter syndrome (Mucopolysaccharidosis II, [MPS II]); Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2014-004804-31-DE
• Lead Sponsor: Shire Human Genetic Therapies, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-08-06
• Last Update Posted: 13 December 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 20

Inclusion Criteria

Patients must meet ALL of the inclusion criteria for his group in order to enroll in the trial.
1. The patient is male.
2. The patient is Elaprase-naïve at study entry.
3. The patient must have a documented diagnosis of MPS II. Of the 3 criteria below, the
combinations (3a AND 3b) or (3a AND 3c) will be accepted as diagnostic of MPS II:
a. The patient has a deficiency in I2S enzyme activity of =10% of the lower limit of
the normal range as measured in plasma, fibroblasts, or leukocytes (based on the
reference laboratory’s normal range).
AND
b. The patient has a documented mutation in the I2S gene.
OR
c. The patient has a normal enzyme activity level of one other sulfatase as measured
in plasma, fibroblasts, or leukocytes (based on the normal range of measuring
laboratory).
4. The patient will be <6 years of age at the start of Elaprase treatment.
5. The patient, patient’s parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved
informed consent form after all relevant aspects of the study have been explained and
discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the
patient’s assent, as relevant, must be obtained
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients who meet any of the following criteria are not eligible for enrollment into this study.
1. The patient has received treatment with any investigational drug or device within the 30 days prior to study entry.
2. The patient has received or is receiving treatment with idursulfase-IT.
3. The patient has received growth hormones, a cord blood infusion, or a bone marrow
transplant at any time.
4. The patient has received blood product transfusions within 90 days prior to Screening.
5. The patient is unable to comply with the protocol as determined by the Investigator.
6. The patient has a history of severe or life-threatening hypersensitivity to the active substance or to any of the excipients if hypersensitivity is not controllable.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified